The CAR-T therapy space has remained in investor focus in 2017, courtesy of immense potential. The latest annual conference of the American Society of Hematology (ASH) witnessed pharma big wigs, including Novartis and Gilead reporting data on their CAR-T therapy candidates.
The FDA has already approved Swiss giant Novartis AG’s NVS breakthrough gene transfer treatment, Kymriah (tisagenlecleucel) for the treatment of patients upto 25 years of age with B-cell precursor acute lymphoblastic leukemia (ALL) that is refractory or in second or later relapse stage. Novartis currently carries a Zacks Rank #3 (Hold). You can see the complete list of today’s Zacks #1 Rank (Strong Buy) stocks here.
Gilead Sciences, Inc. GILD acquired Kite Pharma and subsequently obtained FDA approval of Yescarta (axicabtagene ciloleucel), the latter’s chimeric CAR-T therapy candidate. To further strengthen its position in this space, Gilead Sciences and newly acquired cell therapy subsidiary — Kite Pharma — recently signed an agreement to acquire Cell Design Labs, Inc. for $ 567 million.
Gilead will also acquire 12.2% Cell Design Labs’ shares which are owned by Kite Pharma. Cell Design, is a leading cell-based therapy developer through synNotch and Throttle technology platforms. These technological platforms will enhance Gilead’s cellular therapy research efforts. The company’s lead pre-clinical candidate is instrumental in treating multiple myeloma.
Meanwhile, Kite Pharma announced results from the ongoing phase I/II ZUMA-3 study of KTE-C19, a CD19 CAR-T cell therapy for the treatment of adult patients with relapsed or refractory ALL. 71% of ALL patients who received a single infusion of KTE-C19 achieved complete tumor remission (complete remission (CR) or CR with incomplete hematological recovery). Gilead currently carries a Zacks Rank #3.
Juno Therapeutics, Inc. JUNO and partner Celgene Corp. (CELG) announced additional data from the TRANSCEND study on pipeline candidate JCAR017 (lisocabtagene maraleucel; liso-cel) in patients with relapsed or refractory (r/r) aggressive B-cell non-Hodgkin lymphoma. Data from the trial showed at dose level 2, 74% of patients with the aforementioned indication showed an overall response rate (ORR) and 68% CR rate at three months in core group.
Additionally, 50% of patients showed CR at six months. While the data was encouraging, investors expected far better ORR rates leading to a significant decline in Juno’s shares. A biologics license application filing is expected to be completed in the second half of 2018 and a tentative approval is expected by 2018-end. Juno currently carries a Zacks Rank #3.
bluebird bio, Inc. BLUE and partner Celgene also announced results from the ongoing CRB-401 phase I clinical study on investigational anti-B-cell maturation antigen CAR-T cell therapy, bb2121. The data showed ORR of 94% in patients in active dose cohorts (doses above 50 x 106 CAR + T-cells) and CR rate of 56% observed in patients in active dose cohorts. bluebird currently carries a Zacks Rank #3.
Meanwhile, Novartis submitted a supplemental Biologics License Application to the FDA for Kymriah suspension for intravenous infusion for the treatment of adult patients with r/r diffuse large B-cell lymphoma (DLBCL) who are ineligible for autologous stem cell transplant (ASCT). Moreover, in November, the company submitted an application to the European Medicines Agency for Kymriah for the treatment of adult patients with r/r DLBCL who are ineligible for ASCT, and for children and young adults with r/r B-cell ALL.
Kymriah was given Breakthrough Therapy designation for r/r DLBCL which, if approved, will be the second indication for Kymriah. The company recently announced results from the pivotal JULIET study which showed sustained complete responses at six months with Kymriah suspension for intravenous infusion, in adult patients with a difficult-to-treat cancer- r/r DLBCL. In the study at six months, 30% of patients treated with Kymriah were in complete response. The relapse-free probability at six months after first response was 74%.
Given the immense potential of the therapy, investors are likely to keep an eye on this space in the days ahead as well.
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